A Study to Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181])

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

65 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-07-01

Study Completion Date

2024-08-07

Brief Summary

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This pre-screening study is designed to determine potential eligibility of adults with classic CAH due to 21-hydroxylase deficiency (21-OHD) for participation in the CAH-301 \[NCT04783181\] gene therapy trial with BBP-631.

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Detailed Description

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Adult participants diagnosed with classic CAH due to 21-OHD and who are taking glucocorticoids as treatment for CAH will undergo assessments to determine their potential eligibility for participation in the adult CAH-301 treatment trial with BBP-631.

Conditions

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Congenital Adrenal Hyperplasia

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Overall Study

Participants will undergo antibody and genetic screen, initial pre-screening, eligibility monitoring and eligibility confirmation for potential transition to the treatment trial center once a treatment assignment in the study CAH-301 becomes available. Participation in Study CAH-300 is permitted to continue until treatment assignment is available in the treatment trial or unless, during eligibility monitoring or eligibility

Pre-Screening

Intervention Type OTHER

No intervention given

Interventions

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Pre-Screening

No intervention given

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Adult male and non-pregnant females with classic CAH (simple virilizing or salt-wasting) due to 21-OHD
* Screening/baseline 17-OHP levels \> 5-10 × ULN
* Is on a daily regimen of glucocorticoid
* Naïve to prior gene therapy or AAV-mediated therapy